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Retinoid Therapy for Neuroblastoma
Retinoids are chemicals that are related to vitamin A. They are known as differentiating agents because they are thought to help some cancer cells mature (differentiate) into normal cells.
In children with high-risk neuroblastoma, treatment with a retinoid called 13-cis-retinoic acid (isotretinoin) reduces the risk of the cancer coming back after high-dose chemotherapy and stem cell transplant. Most doctors now recommend 6 months of 13-cis-retinoic acid after the transplant. This drug is taken as a capsule, twice a day for 2 weeks, followed by 2 weeks off.
Researchers are now trying to develop more effective retinoids and to define the exact role of this approach in treating neuroblastoma.
Possible side effects
The most common side effect of 13-cis-retinoic acid is dry and cracked lips. Dry skin or eyes are also possible, as are nosebleeds, muscle and joint pains, and changes in the nails.
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The American Cancer Society medical and editorial content team
Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as editors and translators with extensive experience in medical writing.
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National Cancer Institute. Neuroblastoma Treatment (PDQ). 2020. Accessed at https://www.cancer.gov/types/neuroblastoma/hp/neuroblastoma-treatment-pdq on April 9, 2021.
Park JR, Hogarty MD, Bagatell R, et al. Chapter 23: Neuroblastoma. In: Blaney SM, Adamson PC, Helman LJ, eds. Pizzo and Poplack’s Principles and Practice of Pediatric Oncology. 8th ed. Philadelphia Pa: Lippincott Williams & Wilkins; 2021.
Pinto NR, Applebaum MA, Volchenboum SL, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol. 2015: 30;3008-3017.
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Last Revised: April 28, 2021
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